study design

April 6, 2020: Convening Pain Management Expertise in the Patient-Centered Outcomes Core: An Interview With Dr. Kevin Weinfurt

Posted on by Gina Uhlenbrauck

The Collaboratory recently interviewed Kevin Weinfurt, PhD, co-chair of the Patient-Centered Outcomes Core working group, to hear about the Core’s plans for supporting the PRISM Demonstration Projects. In this video, Dr. Weinfurt says one challenge for embedded trials of pain is in obtaining high-quality, complete patient-reported outcome data on pain intensity and functioning. Along with other national initiatives focused on pain research, such as the NIH-DoD-VA Pain Management Collaboratory, a chief task of the Core will be to look at efforts to harmonize pain outcomes across studies.

“The Core serves as a convener of expertise in pain management within the Collaboratory. We keep an eye on issues across the Demonstration Projects that might also be of interest to the broader pragmatic trial community.”

Read more about the work of the Patient-Centered Outcomes Core, and view other interviews about the HEAL Initiative and PRISM Demonstration Projects.

March 27, 2020: Living Textbook Grand Rounds Series: Part 3-Tips for Putting Together a Successful PCT Grant Application (Wendy Weber, ND, PhD, MPH)

Posted on by Gina Uhlenbrauck

Speaker

Wendy Weber, ND, PhD, MPH
Branch Chief, Clinical Research Branch
Division of Extramural Research
National Center for Complementary and Integrative Health (NCCIH)

Topic

Tips for Putting Together a Successful PCT Grant Application

Keywords

Pragmatic clinical trials; Embedded PCTs; Healthcare systems; NIH; Scientific officers; Funding opportunity announcement; Request for application

Key Points

  • Investigators interested in developing a compelling grant application for a pragmatic clinical trial (PCT) should consider these tips:
    • First, confirm that a PCT is the best study design to answer your research question.
    • From the NIH’s 27 Institutes and Centers, find the one that matches your disease area, organ system, or stage of life—and talk with the program official.
    • Also find the right funding opportunity announcement (FOA) and tailor your application to address all the FOA-specific instructions and review criteria.
  • Do not assume that the study panel is going to understand what pragmatic means; they may have a completely different definition. It is important that you get on the same page early in the application.
  • In your application, make sure you justify the research, include pilot data, address potential overlaps, reduce complexity, ensure the aims will advance the field, choose expert personnel for a multidisciplinary team, link data collection and analysis to aims, and justify the use of multiple sites and sample size.

Discussion Themes

For some urgent clinical problems, a PCT may be the only study design that can answer the research question.

Phased awards can be helpful. Determine the key elements of your trial to test in the planning phase. It’s important to “fail quickly”—not in the implementation phase. Applicants should identify milestones in the planning phase that will help convince reviewers their study will be feasible.

Because PCTs take place in changing conditions and real-world healthcare settings, they are not necessarily easier to implement than a traditional efficacy trial.

For more information, visit the Living Textbook’s Developing a Compelling Grant Application.

Tags
#pctGR, @Collaboratory1

March 31, 2020: Engagement in PCTs: Considerations from the Collaboratory’s Ethics and Regulatory Core

Posted on by Gina Uhlenbrauck

A new document from the Ethics and Regulatory Core is available that provides considerations around determining which individuals or groups are engaged in research in pragmatic clinical trials (PCTs). Developed for investigators designing and conducting PCTs as well as institutional review boards overseeing them, the document introduces these questions in relation to research subjects, study team members, and service providers:

  • Which individuals/groups are included in the research?
  • Are these individuals/groups research subjects, study team members, or service providers?
  • Why does it matter how the individuals/groups are categorized for the research?

Download Engagement in Research for Pragmatic Clinical Trials.

 

March 30, 2020: New Living Textbook Chapter Describes Ways to Monitor Intervention Fidelity and Adaptations During the Conduct of ePCTs

Posted on by Gina Uhlenbrauck

The new Monitoring Intervention Fidelity and Adaptations chapter of the Living Textbook was developed to introduce how to evaluate changes that may be encountered while conducting an embedded pragmatic clinical trial (ePCT). For example, a health system might experience competing clinical initiatives; turnover in leadership, clinicians, or staff; changes in technologies; new clinical practice guidelines; or regulatory changes.

The chapter offers strategies for study teams to anticipate, monitor, and document adaptations to the intervention in order to support study analysis and set the stage for dissemination and implementation of successful interventions in other healthcare settings. The chapter was developed by experts from the Collaboratory’s Health Care Systems Interactions Core, along with principal investigators conducting ePCT Demonstration Projects.

Topics include:

  • Anticipating changes that might impact intervention fidelity
  • Frameworks to assist in monitoring fidelity and adaptations
  • Strategies and case examples from the NIH Collaboratory Demonstration Projects
  • Pointers to additional resources

We encourage you to explore this new content.

 

March 17, 2020: Cheat Sheet on the Intraclass Correlation Coefficient

Posted on by Karen Staman

The NIH Collaboratory Biostatistics and Study Design Core has created an Intraclass Correlation Coefficient (ICC) Cheat Sheet to provide an introductory description of the ICC, which is important for the design and analysis of cluster-randomized trials.

“The intraclass correlation coefficient (ICC) is a descriptive statistic that describes the extent to which outcomes 1) within each cluster are likely to be similar or 2) between different clusters are likely to be different from each other, relative to outcomes from other clusters. The ICC is an important tool for cluster-randomized pragmatic trials because this value helps determine the sample size needed to detect a treatment effect.” —from the ICC Cheat Sheet

The tool is a 2-page handout that can be used in trainings or classes regarding pragmatic clinical trials involving cluster randomization.

For more on the ICC, see the Intraclass Correlation section in the Living Textbook or this in-depth working document on the ICC from the Biostatistics and Study Design Core. If you have questions, feedback or suggestions regarding this tool, please contact us at nih-collaboratory@dm.duke.edu.

March 12, 2020: Transforming Our Understanding of How to Conduct Research in a Real-World Setting

Posted on by Gina Uhlenbrauck

In a recent video interview, Dr. Helene Langevin, director of the National Center for Complementary and Integrative Health at the NIH, discusses the important work of the NIH Collaboratory in sharing knowledge and lessons learned as well as in harnessing expertise across the 19 Demonstration Projects it supports. “It is very important that we understand how research can be successfully implemented and tested in real-world clinical practice.”

 

 

For more interviews about the HEAL and PRISM projects, check out the February 21 news story.

March 6, 2020: Creating a Learning Health System Through Randomization (Leora Horwitz, MD, MHS)

Posted on by Gina Uhlenbrauck

Speaker

Leora Horwitz, MD, MHS
Associate Professor, Population Health and Medicine
Director, Center for Healthcare Innovation and Delivery Science, NYU Langone Health
Director, Division of Healthcare Delivery Science, Department of Population Health, NYU Grossman School of Medicine

Topic

Creating a Learning Health System Through Randomization

Keywords

Embedded research; Electronic health records; Randomization; Quality improvement (QI); Learning health systems

Key Points

  • Randomized QI projects are needed in order to know whether such system-level programs/interventions are effective.
  • The best candidates for QI randomization projects have a high volume of cases and short-term outcomes that are already being collected.
  • These randomized QI projects were developed in collaboration with the frontline care providers and staff to ensure seamless implementation with no additional burden. Also essential was buy-in from senior-level administration.

Discussion Themes

Statistical significance is not always the right threshold for decision making in a health system.

What amount of resources are needed to do these projects (eg, researcher/analysts, project managers)? Is the work self-sustaining?

Disappointing QI findings are important to know, so that ineffective activities can be discontinued.

Read more about this project in Creating a Learning Health System through Rapid-Cycle, Randomized Testing (Horwitz et al., New Engl J Med, Sep 2019).

Tags
#pctGR, @Collaboratory1

March 10, 2020: Supporting the New PRISM NIH Collaboratory Trials Addressing Pain Management Within Care Delivery

Posted on by Gina Uhlenbrauck

In a new video interview, Dr. Lesley Curtis, chair of the NIH Collaboratory Steering Committee, describes the importance of the four PRISM NIH Collaboratory Trials, which are directly addressing the U.S. opioid crisis and exploring fundamental questions of pain management using nonpharmacologic treatments. “We are particularly excited about the answers that will come out of these trials,” she says. Now in their UG3 planning year, the PRISM investigators are strongly encouraged to take full advantage of the Collaboratory’s resources, such as the significant expertise found across the Core Working Groups.

 

 

For more interviews about the HEAL and PRISM projects, check out the February 21 news story.

February 28, 2020: Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts

Posted on by Gina Uhlenbrauck

Speakers

Vincent Mor, PhD
Florence Pirce Grant University Professor
Department of Health Services Policy & Practice
Brown University School of Public Health

Leah Tuzzio, MPH
Research Associate
Kaiser Permanente Washington Health Research Institute

Jeffrey (Jerry) G. Jarvik MD MPH
Professor, Radiology, Neurological Surgery and Health Services
Adjunct Professor, Pharmacy and Orthopedics & Sports Medicine
University of Washington

Topic

Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts

Keywords

Embedded research; Implementation; Healthcare systems; Nursing homes; Radiology reports; Electronic health records; Data collection; Pragmatic clinical trials

Key Points

  • Early engagement between study teams and health system leaders and staff can help ease the embedded intervention into the clinical workflow. Consider, for example, who will deliver the intervention and how difficult it will be to implement.
  • In the pilot phase, show that you can implement the intervention effectively. For example, demonstrate that you can collect the outcome data you will need within the pilot’s time frame. Ensure that your study questions are important to the partner healthcare system.

Discussion Themes

It is essential to anticipate, identify, and make a plan to address changes in the healthcare system as your trial is being conducted.

Among the NIH Collaboratory Trials, many have good measures of how much site-to-site variation there is, which helps to understand what the preconditions are for high levels of implementation.

Use the pilot study to maximize acceptability, maintain affordability, and consider the scalability of the intervention.

These topics and more are on the Living Textbook of Pragmatic Clinical Trials. Plan to hear the next topics in the Living Textbook Grand Rounds series.

Tags
#pctGR, @Collaboratory1

February 28, 2020: Meeting Minutes from NIH Collaboratory’s Ethics and Regulatory Core Discussions with the PRISM NIH Collaboratory Trials

Posted on by Gina Uhlenbrauck

Meeting minutes and supplementary materials are available that summarize discussions related to the ethics and regulatory issues associated with each of the UG3 PRISM NIH Collaboratory Trials. These discussions, which took place by teleconference, included representation from study principal investigators and study teams, members of the NIH Collaboratory Ethics and Regulatory Core, NIH staff, and NIH Collaboratory Coordinating Center personnel as well as some IRBs responsible for oversight of the projects.